Autism Spectrum DisorderPhase 1NCT05602116
ASC - Autism Study
A Phase 1 study testing Adipose tissue derived mesenchymal stromal/stem cells (Cell2Cure®) in people with Autism Spectrum Disorder and Gastrointestinal Dysfunction, run by Cell to Cure ApS at 1 site in Denmark.
Trial summary
The ASC - Autism Pilot Study is a single center randomized open dose titrating phase I clinical intervention pilot trial with the aim of investigating safety and treatment effect of an allogeneic adipose tissue derived mesenchymal stromal cell product (C2C_ASC) in children with autism spectrum disorder (ASD) and… Read more about this study
- Who is it for?
- Ages 6 to 14 years
- Conditions the study is for
- Autism Spectrum Disorder, Gastrointestinal Dysfunction, Leaky Gut Syndrome
- What is being tested?
- Adipose tissue derived mesenchymal stromal/stem cells (Cell2Cure®)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteDenmark
- Study dates
- 9 Jun 2023to 20 Sep 2024
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 14 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Diagnosis of autism spectrum disorder.
- Ongoing gastrointestinal symptoms or previous gastrointestinal symptoms which disappeared or were reduced after dietary changes.
- Age 6 to 14 years.
Who cannot join
- Known genetic syndrome or pathogenic mutation or copy number variation associated with autism spectrum disorder.
- Known CNS-infection (now or previously) and/or HIV positivity.
- Primary immunodeficiency disorder or autoimmune cytopenia.
- Current treatment with cytotoxic drugs or systemic administered glucocorticoids and/or immunosuppressive therapy or other antiinflammatory medication (except nonsteroidal anti-inflammatory drugs).
- Epilepsy or known seizure disorder (now or previously).
Trial locations and contacts
1 site in 1 country.
Denmark1 site
Department of Child and Adolescent Psychiatry, Psychiatry Clinic South, Aalborg University Hospital, Denmark
Aalborg
About this study
Official title: Effect of Adipose Tissue Derived Mesenchymal Stromal Cells on Autism and Leaky Gut Syndrome. A Phase I Pilot Study.
The ASC - Autism Pilot Study is a single center randomized open dose titrating phase I clinical intervention pilot trial with the aim of investigating safety and treatment effect of an allogeneic adipose tissue derived mesenchymal stromal cell product (C2C_ASC) in children with autism spectrum disorder (ASD) and gastrointestinal symptoms.
Read the detailed description
The ASC - Autism Pilot Study will be performed at the Child and Adolescent Psychiatry, Aalborg University Hospital, Denmark. 10 children aged 6-14 years with ASD and gastrointestinal symptoms will be enrolled through the Child and Adolescent Psychiatry, Psychiatry Clinic South, Aalborg University Hospital, Denmark.
The participants will randomly be divied into two treatment groups:
- participant 1-5 will be treated with 1 x 1000000 C2C_ASCs/kg body weight
- participant 6-10 will be treated with 2 x 1000000 C2C_ASCs/kg body weight
C2C_ASC treatment:
The C2C_ASCs product will be diluted in 50 ml isotonic saline and infused in an arm vein within 15 min. In both groups there will for each participant be a week observation for SAE's related to the cell treatment before the next participant will be treated, if no SAE's have been detected.
The investigators hypothesize that the connection between gastrointestinal symptoms/leaky gut syndrome, increase local gastrointestinal and systemic elevated immunological and inflammatory activity, bacterial toxins in the blood and symptoms of ASD in children with symptoms of leaky gut syndrome can be reduced or normalized by modulating the immunological activity and inflammation by treatment with mesenchymal stromal cells.
- Conditions
- Autism Spectrum Disorder, Gastrointestinal Dysfunction, Leaky Gut Syndrome, Mesenchymal Stromal Cells, Inflammatory Bowel Diseases
- Being tested
- Adipose tissue derived mesenchymal stromal/stem cells (Cell2Cure®)
- Keywords
- Gastrointestinal microbiota, Gut-brain axis, Immunomodulation
- Medical subject headings
- Autism Spectrum Disorder, Inflammatory Bowel Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Adipose tissue derived mesenchymal stromal/stem cells (Cell2Cure®)Drug
The Investigational Product is a C2C_ASC cell product (Cell2Cure®, Cell2Cure Aps, Denmark).
C2C_ASC is an advanced therapy investigational medicinal product (ATIMP) manufactured from abdominal adipose tissue derived mesenchymal stromal cells (ASC) from healthy donors. C2C_ASC is aseptically procured and manufactured according to tissue law and GMP by Cell2Cure ApS or Cardiology Stem Cell Center, Rigshospitalet, Copenhagen, Denmark, using manual isolation of cells from abdominal fat tissue, xeno-free cell expansion in automated closed bioreactor systems and cryopreservation of the final product.
The active substance is the in vitro expanded ASCs. The final product, C2C_ASC, is provided as a cryopreserved suspension of 50 million ASCs per ml with a total volume of 1,3 ml per vial. The excipient is CryoStor10 (Biolife Solutions), holding 10% DMSO.
| Study group | Role | What they receive |
|---|---|---|
| 1 x 1000000 C2C_ASCs/kg body weight | Experimental | Trial participant 1 - 5 will be treated with 1 x 1000000 C2C_ASCs/kg body weight Receives: Drug: Adipose tissue derived mesenchymal stromal/stem cells (Cell2Cure®) |
| 2 x 1000000 C2C_ASCs/kg body weight | Experimental | Trial participant 6 - 10 will be treated with 2 x 1000000 C2C_ASCs/kg body weight Receives: Drug: Adipose tissue derived mesenchymal stromal/stem cells (Cell2Cure®) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety of C2C_ASC (Adverse Events)
Safety of C2C_ASC within the 12 weeks follow-up period: Adverse event (AE), serious adverse event (SAE), suspected unexpected serious adverse reaction (SUSAR) and development safety update report (DSUR).
Measured: 12 weeks
Secondary outcomes (2)
Changes in autism spectrum disorder
Difference from baseline to follow-up within the group treated with C2C_ASC:
Changes in autism spectrum disorder score test (questionnaire).
[Raw scores converting to standard scores and then generates five major domain composite scores: communication, daily living skills, socialization, adaptive behavior composite and motor. For these domains a standard score of 100 is the mean with a standard deviation of 15. This means that a score of 100 is similar to the typical population of the same age. Scores in the range of 86 and above are considered adequate. Scores in the range of 85 and below indicates a significant skill deficit when comparing with the typical population of the same age.
High scores: standard scores of 130-140 Moderately high scores: standard scores of 115-129 Adequate scores: standard scores of 86-114 Moderately low scores: standard scores of 71-85 Low scores: standard scores of 20-70]
Measured: 12 weeks
Changes in gastrointestinal symptoms
Difference from baseline to follow-up within the group treated with C2C_ASC:
Changes in The Infant Gastrointestinal Symptom Questionnaire (questionnaire).
[A 0-100 scale where higher scores indicate better irritable bowel syndrome specific quality of life.]
Measured: 12 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Nov 2022
- Study start9 Jun 2023
- Primary completion28 Jun 2024
- Study completion20 Sep 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Autism Spectrum DisorderStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Model description
- Open dose titrating with two groups in extension of each other (thus not categorically parallel).
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- Cell to Cure ApS
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Jens Kastrup, Study director, Cell to Cure ApS
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05602116
- Sponsor's protocol ID
- ASC II
- Status last verified
- Sep 2024
- First submitted
- 23 Sep 2022
- Last updated
- 26 Sep 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 26 Sep 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effect of Adipose Tissue Derived Mesenchymal Stromal Cells on Autism and Leaky Gut Syndrome. A Phase I Pilot Study.
- ClinicalTrials.gov ID
- NCT05602116
- Acronym / sponsor ID
- ASC II
- Conditions
- Autism Spectrum Disorder, Gastrointestinal Dysfunction, Leaky Gut Syndrome, Mesenchymal Stromal Cells, Inflammatory Bowel Diseases
- Keywords
- Gastrointestinal microbiota, Gut-brain axis, Immunomodulation
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- Cell to Cure ApSIndustry (pharma, biotech, devices)
- Study start
- 9 Jun 2023 (actual)
- Primary completion
- 28 Jun 2024 (actual)
- Study completion
- 20 Sep 2024 (actual)
- First posted
- 1 Nov 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The ASC - Autism Pilot Study is a single center randomized open dose titrating phase I clinical intervention pilot trial with the aim of investigating safety and treatment effect of an allogeneic adipose tissue derived mesenchymal stromal cell product (C2C_ASC) in children with autism spectrum disorder (ASD) and gastrointestinal symptoms.
Detailed description
The ASC - Autism Pilot Study will be performed at the Child and Adolescent Psychiatry, Aalborg University Hospital, Denmark. 10 children aged 6-14 years with ASD and gastrointestinal symptoms will be enrolled through the Child and Adolescent Psychiatry, Psychiatry Clinic South, Aalborg University Hospital, Denmark.
The participants will randomly be divied into two treatment groups:
- participant 1-5 will be treated with 1 x 1000000 C2C_ASCs/kg body weight
- participant 6-10 will be treated with 2 x 1000000 C2C_ASCs/kg body weight
C2C_ASC treatment:
The C2C_ASCs product will be diluted in 50 ml isotonic saline and infused in an arm vein within 15 min. In both groups there will for each participant be a week observation for SAE's related to the cell treatment before the next participant will be treated, if no SAE's have been detected.
The investigators hypothesize that the connection between gastrointestinal symptoms/leaky gut syndrome, increase local gastrointestinal and systemic elevated immunological and inflammatory activity, bacterial toxins in the blood and symptoms of ASD in children with symptoms of leaky gut syndrome can be reduced or normalized by modulating the immunological activity and inflammation by treatment with mesenchymal stromal cells.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- Open dose titrating with two groups in extension of each other (thus not categorically parallel).
| Arm | Type | Receives |
|---|---|---|
| 1 x 1000000 C2C_ASCs/kg body weight | Experimental | Drug: Adipose tissue derived mesenchymal stromal/stem cells (Cell2Cure®)Trial participant 1 - 5 will be treated with 1 x 1000000 C2C_ASCs/kg body weight |
| 2 x 1000000 C2C_ASCs/kg body weight | Experimental | Drug: Adipose tissue derived mesenchymal stromal/stem cells (Cell2Cure®)Trial participant 6 - 10 will be treated with 2 x 1000000 C2C_ASCs/kg body weight |
Who may qualify
- Ages
- 6 to 14 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- Diagnosis of autism spectrum disorder.
- Ongoing gastrointestinal symptoms or previous gastrointestinal symptoms which disappeared or were reduced after dietary changes.
- Age 6 to 14 years.
Key exclusion criteria (5 listed)
- Known genetic syndrome or pathogenic mutation or copy number variation associated with autism spectrum disorder.
- Known CNS-infection (now or previously) and/or HIV positivity.
- Primary immunodeficiency disorder or autoimmune cytopenia.
- Current treatment with cytotoxic drugs or systemic administered glucocorticoids and/or immunosuppressive therapy or other antiinflammatory medication (except nonsteroidal anti-inflammatory drugs).
- Epilepsy or known seizure disorder (now or previously).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Adipose tissue derived mesenchymal stromal/stem cells (Cell2Cure®)The Investigational Product is a C2C_ASC cell product (Cell2Cure®, Cell2Cure Aps, Denmark). C2C_ASC is an advanced therapy investigational medicinal product (ATIMP) manufactured from abdominal adipose tissue derived mesenchymal stromal cells (ASC) from healthy donors. C2C_ASC is aseptically procured and manufactured according to tissue law and GMP by Cell2Cure ApS or Cardiology Stem Cell Center, Rigshospitalet, Copenhagen, Denmark, using manual isolation of cells from abdominal fat tissue, xeno-free cell expansion in automated closed bioreactor systems and cryopreservation of the final product. The active substance is the in vitro expanded ASCs. The final product, C2C_ASC, is provided as a cryopreserved suspension of 50 million ASCs per ml with a total volume of 1,3 ml per vial. The excipient is CryoStor10 (Biolife Solutions), holding 10% DMSO. | Drug | 1 x 1000000 C2C_ASCs/kg body weight, 2 x 1000000 C2C_ASCs/kg body weight |
Primary outcome
Safety of C2C_ASC (Adverse Events)
Time frame: 12 weeks
Safety of C2C_ASC within the 12 weeks follow-up period: Adverse event (AE), serious adverse event (SAE), suspected unexpected serious adverse reaction (SUSAR) and development safety update report (DSUR).
2 secondary outcomes
Changes in autism spectrum disorder
Time frame: 12 weeks
Changes in gastrointestinal symptoms
Time frame: 12 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Sep 2024.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Jens Kastrup study director
Cell to Cure ApS
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Adipose tissue derived mesenchymal stromal/stem cells (Cell2Cure®) in people with Autism Spectrum Disorder and Gastrointestinal Dysfunction, run by Cell to Cure ApS at 1 site in Denmark.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 26 Sep 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Denmark. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety of C2C_ASC (Adverse Events) (time frame: 12 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Sep 2024.